Efforts to slow down the aging process are not just about wrinkles. Getting older is also the leading risk factor for devastating diseases such as cancer, heart disease, Alzheimer's and dementia.
In a paper published this week in the journal Natural Medicine, scientists reveal a new CRISPR / Cas9 genome-editing therapy was able to slow the rapid aging associated with Hutchinson-Gilford progeria syndrome in laboratory mice.
"Aging is a complex process in which cells begin to lose their functionality, so it is critical for us to find effective ways to study the molecular drivers of aging" says senior author and Salk professor Juan Carlos Izpisua Belmonte in a statement. Progeria, he says, is an "ideal aging model" because they are able to intervene and observe results in a short period of time.
Progeria is caused by a mutation of the LMNA gene. This gene produces the lamin A and lamin C proteins in the cell. But for those with the disease, the LMNA gene instead creates progerin ̵
The scientists' goal was to "disrupt" progerin production using CRISPR / Cas9-targeted therapy
Cas9 is like a molecular surgeon. The protein is coupled with a synthetic RNA sequence that matches and attracts the malfunctioning DNA strand and cuts it out (i.e., gene-editing). In this case, Salk researchers used an adeno-associated virus (AAV) – which causes a very low immune response – to deliver the Cas9 caravan, including two "guide" RNAs and a "reporter" gene, which lets them know
Two months after gene-editing, the mice were stronger and were about as active as average healthy mice. They also showed improvements among progeria-specific conditions, including arterial blood vessel damage and low heart rate. Overall, those treated saw a life span increase by about 25 percent
Given these results, scientists believe CRISPR / Cas9 gene-editing therapy shows a lot of promise for targeting the causes of cell degeneration caused by aging in humans. ] "This is the first time that gene therapy treatment has been applied to treat progeria syndrome," says Izpisua Belmonte. "This is an exciting advancement."